On 14 November 2007, orphan designation (EU/3/07/486) was granted by the European Commission to Centre Hospitalier Universitaire de Nantes, France, for adenovirus associated viral vector serotype 4 containing the human RPE65 gene for treatment of retinitis pigmentosa.
The sponsorship was transferred to Horama SAS, France, in May 2015.
In November 2016 Horama SAS changed name to Horama SA.
The sponsor’s name was changed from Horama SAS to Coave Therapeutics and the address was updated in August 2022.
Adenovirus associated viral vector serotype 4 containing the human RPE65 gene
Treatment of retinitis pigmentosa
|Orphan designation status||
|EU designation number||
|Date of designation||
Documents related to this orphan designation evaluation
EU/3/07/486: Public summary of positive opinion for orphan designation of adenovirus associated viral vector serotype 4 containing the human RPE65 gene for the treatment of retinitis pigmentosa (PDF/122.18 KB)
First published: 02/07/2008
Last updated: 27/05/2015
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: